The Next Decade In Gene Therapy Innovation â 6 Critical Questions (And Answers)
By Kyle M. OâNeil and Brendan J. Wang, Back Bay Life Science Advisors
As the gene therapy field has tackled delivery vectors as described in “An Analysis Of The Gene Therapy Viral Vector Landscape,” several clinical and commercial questions lowered expectations for gene therapy sales, leading sell-side forecasts to fall more than 50% since 2018.
1 However, more than $6.5 billion in 2026 projected gene therapy sales remain across Duchenne muscular dystrophy (DMD), hemophilia A/B, sickle cell disease, Fabry disease, Huntington’s disease, and Leber congenital amaurosis type 10 (LCA10) alone, putting pressure on industry-leading gene therapy companies to deliver.
Published: May 21, 2021 By Alex Keown
Biopharma and life sciences companies strengthen their leadership teams and board with these Movers & Shakers.
AavantiBio – Jessie Hanrahan was named chief regulatory officer of AavantiBio. Hanrahan will oversee global regulatory affairs for AavantiBio’s diversified pipeline of gene therapy programs targeting rare diseases with significant unmet medical needs. Most recently, she was Vice President of Regulatory Science at bluebird bio, Inc. Prior to bluebird bio, Hanrahan held positions of increasing responsibility at Genzyme Corp. where she worked across the oncology, multiple sclerosis and rare disease therapeutic areas. Earlier in her career, Hanrahan worked at Boston Scientific Corporation.
Oncolytic Virus Cancer Therapy Pipeline Analysis: 85+ Key Pipeline Therapies and 45+ Key Pharma Competitors to Look Out for in the Oncolytic Virus Cancer Therapy Pipeline Landscape sandiegosun.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from sandiegosun.com Daily Mail and Mail on Sunday newspapers.
Neurocrine Biosciences Reports First Quarter 2021 Financial Results
INGREZZA® (valbenazine) First Quarter 2021 Net Product Sales of $230 Million with Approximately 43,300 TRx
Initiated Pediatric Phase III Registrational Program of Crinecerfont for the Treatment of Classic Congenital Adrenal Hyperplasia
Initiated Phase II Study of NBI-827104 in Essential Tremor
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SAN DIEGO, May 5, 2021 /PRNewswire/ Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced its financial results for the first quarter ended March 31, 2021 and provided revised full-year 2021 financial expense guidance. Our first quarter results reflect a lower than normal refill rate per patient due to the typical seasonal payor dynamics for INGREZZA that were exacerbated by COVID. Importantly, we did not see an increase in discontinuations and exited the quarter with more patients on INGREZZA versus the prior quarter. New patient starts did pick up late